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Thursday, March 31. 2016

Believe it or not, there is a battle underway in the U.S. over whether terminally ill patients who have exhausted all government-approved treatments have the right to try experimental drugs. You might think that in a country founded on the principles of limited government and individual rights, citizens would be free to choose any health remedy in a last-ditch effort to save their own lives. But that’s not the way it is: Congress gave the Food and Drug Administration (FDA) exclusive power to approve drugs, and the FDA believes that even people who are dying must be protected from unproven therapies.

Heartbreaking stories about dying patients pleading for access to promising but not-yet-approved drugs forced the FDA to act years ago. The FDA created an “expanded access” program in 1987 enabling patients to apply for special permission to try investigational drugs. But there were complaints that the FDA wasn’t giving all patients a fair hearing. That led to the Food and Drug Administration Modernization Act of 1997 establishing more formal expanded access program rules.

Nearly 20 years later, terminally ill patients and their physicians must still jump through multiple hoops with no reliable way of predicting the outcome. To have a chance, the patient must obtain the approval of a physician and an institutional review board (IRB). The FDA estimates it takes physicians on average 100 hours to complete the forms. (The FDA has been showing a draft form that it says can be completed in just 45 minutes since February 2015, but more than one year later the miracle form still hasn’t been released.) The IRB is a local committee established to protect research subjects from physical and psychological harm. Even if the physician and IRB recommend approving the patient’s application, the FDA reserves the right to say “no.”

The deck is clearly stacked against dying patients who must find physicians willing to work many extra hours with no guarantee of success.

The FDA points to the expanded access program as proof that it is compassionate. But for some patients the program has failed spectacularly. Kianna Karnes, a mother of four children suffering from kidney cancer, requested access to two promising drugs. She didn’t qualify to participate in clinical trials. Her family lobbied on her behalf for months and finally the FDA approved her application. But it was too late: Kianna Karnes died later that day. While the FDA’s defenders complain that activists are giving terminally ill patients false hope, both of the drugs that Karnes sought access to were eventually approved by the FDA for treating kidney cancer.

How did a federal agency acquire the power to decide who lives and who dies? The FDA is an ominous example of how a government agency can gradually accumulate the power to deprive citizens of their rights and hobble entire industries.

Over most of U.S. history, citizens were free to take whatever medicines they wished. Ironically, it wasn’t until effective and often life-saving drugs appeared in the 20th century that strict regulations were introduced.

Many federal drug regulations were created in response to widely reported acts of negligence or wrongdoing. Concerns about the safety of preservatives and other additives led to the Pure Food and Drug Act of 1906. During the mid-1930s, the S.E. Massengill Company introduced a sulfa drug in liquid form. The company tested the drug for safety but forgot to test the solvent. More than 100 patients died from kidney failure. That prompted Congress to pass the Food, Drug, and Cosmetic Act of 1938, a law requiring government approval of new drugs and medical devices. A series of amendments and additional laws expanded the FDA’s power. The 1951 Durham-Humphrey amendment gave the FDA the power to require prescriptions for certain drugs.

The 1962 Kefauver Harris Amendment was the turning point, however. Enacted in response to the thalidomide tragedy in Europe, it required manufacturers to prove not only that new drugs are safe, but that they are effective. This amendment is responsible for adding years of delays and $billions in cost to life-saving drugs. Worse, it led to the misguided notion that if a new drug only cures 5 percent of patients, then it should be denied approval.

Recently, terminally ill patients and their families have been fighting back. “Right to try” laws have been passed in 25 states. These laws have many of the same requirements as the FDA’s expanded access program. The biggest difference is that a state government rather than the FDA is in charge. Right to try laws work because state governments are less conflicted about granting dying patients access to drugs that have not yet been approved, and the FDA is reluctant to challenge states’ rights.

Why is this end run necessary? The FDA has a habit of implementing reforms and creating new programs with one hand, while obstructing and delaying with the other. Darcy Olsen, President of the Goldwater Institute and author of The Right to Try, likens the FDA’s behavior to the famous gag in the Peanuts comic strip in which Lucy holds a football for Charlie Brown to kick, but pulls it away at the very last instant, leaving Charlie sprawled on the ground.

The FDA claims that it “fast tracks” approval of promising drugs. However, that’s misleading: The FDA measures approval from the time that a new drug application (NDA) is submitted, but the NDA is actually the last step in a lengthy process. Companies don’t submit NDAs until the FDA tells them that they’ve met all research, development, and clinical trial requirements. For instance, the FDA claims that it took just six months and 24 days to approve Beleodaq (for treating peripheral T-cell lymphoma), but it actually took over nine years.

A compelling case can be made that some drugs and medical devices should be closely monitored for safety, quality, and accurate labels. Whether federal regulations or industry self-regulation works best is a debate for another time. But one thing is clear: the effectiveness of a medical therapy may vary from one patient to another, and in no case should a government agency’s assessment of effectiveness be allowed to deprive patients, companies, and entire industries of their rights.

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